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Noticias y Eventos

Descubra las novedades en el campo de las patologías oculares hereditarias

Blog operado por el equipo de DBGen que actualiza terapias, noticias y eventos relacionados con las enfermedades hereditarias oculares. Le invitamos a participar en este foro.

Organoids, generating human eyes for therapy

28/06/22 - Gene editing, Research, Therapy,

  Nowadays, most Hereditary Retinal Diseases lack effective treatment. One of the challenges in the development of new therapies is the difficulty to study the functional and physiological effects of the more than 270 genes that are associated with these pathologies. The second challenge is the generation of a reliable model in which to perform a massive screening of potentially...

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DBGen presents and interviews Lluís Montoliu on International Albinism Day

  Today we celebrate International Albinism Day. On this occasion, DBGen interviews Lluís Montoliu, an internationally renowned scientist for his long research career and his relevant contributions to the knowledge of the genetic and molecular basis of albinism. In addition, he is a renowned specialist in gene editing, the generation of animal models of rare diseases, and a highly recognized...

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A GREAT LEAP IN THE TREATMENT OF HEREDITARY PATHOLOGIES OF VISION: GENE THERAPY

Of all the advances that scientific and technological development has brought in the field of hereditary pathologies that affect vision, the most relevant and transcendent for the patient is the possibility of applying treatments for diseases that, until now, were considered incurable. Thirty years ago a new type of therapy was successfully launched: gene therapy. In general terms, gene therapy...

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TwinPE: An improved genome-editing tool that allows inserting entire genes for gene therapy

Researchers at the Broad Institute of Harvard University have just reported in the scientific journal Nature Biotechnology an improved version of prime editing, the highly accurate genome editing method developed in 2019 by the same team. This new version, called twin prime editing (twinPE), allows insertion of DNA fragments of the size of a gene into specific sites of the...

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Novel high-precision gene therapy to correct an eye disease in mice

Researchers from the Seoul National University (Korea) have applied for the first an improved genome-editing methodology, named prime editing to correct the genetic defects of two animal models of human disease: a liver disease (hereditary tyrosinemia) and an eye disease (Leber congenital amaurosis). This high-precision genome-editing tool, developed only two years ago by the team of David R. Liu from...

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A revolutionary CRISPR gene editing technique applied for the first time to a patient with a visual disorder

The patient, treated at the Casey Eye Institute in Portland, Oregon, belongs to a group of 18 people affected with Leber congenital amaurosis. These patients will participate in the BRILLIANCE study, the first clinical trial to determine the safety and efficacy of a therapy based on the CRISPR-Cas9 gene editing technique (AGN-151587, EDIT-101). It is the first example of an...

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